Table 1. Sign of efficacy and MTD in 317 phase 1 oncology trials.

	Trials investigating Conventional cytotoxic agents	Trials investigating molecular targeted agents	Trials investigating combination of both
MTD established
no. (%)	152/201 (75.6) *	38/74 (51.3)	18/42 (42.8)
[95%-CI]	[69.7–81.5]	[39.9–62.7]	[27.8–57.8]
Dose-level associated with DMT
Median	5	5	4
Range	1–13	1–16	1–12
Sign of efficacy
no. (%)	165/201 (82.0)	58/74 (78.3)	31/42 (73.8)
[95%-CI]	[76.7–87.4]	[69.8–87.7]	[60.5–87.7]
Dose-level associated with first sign of efficacy
Median	1	1	1
Range	1–7	1–9	1–3
First sign of efficacy seen at the first dose-level
no. (%)	57/109 (52.2)	15/60 (25.0) *	16/31 (51.6)
[95%-CI]	[42.9–61.6]	[14.0–35.9]	[34.0–69.2]
First sign of efficacy seen at the first 3 dose-levels
no. (%)	81/109 (74.3)	29/60 (48.3) *	19/31 (61.3)
[95%-CI]	[66.1–82.5]	[35.6–60.9]	[44.1–78.4]
First sign of efficacy seen before MTD
no. (%)	91/144 (63.2)	27/44 (61.3)	13/25 (52.0)
[95%-CI]	[55.3–71.0]	[46.9–75.7]	[32.4–71.6]
Sign of efficacy without MTD reached
no. (%)	26/193 (13.4)	10/66 (15.0)	21/32 (65.6) *
[95%-CI]	[8.6–18.3]	[6.5–23.8]	[49.1–82.0]

MTD: dose maximal tolerated dose;

*: p<0.05.

The total of trials within the same treatment categories could differ in the different lines because no data concerning MTD was available in 28 trials and because the first sign of efficacy was not linked to a dose-level in 64 trials.